To summarize insights generated during the preceding four conventions of the European Access Academy (EAA) regarding the interface of patient organizations and medical societies with the evolving European Union (EU) health technology assessment (HTA) process.

In 2022 and 2023 four EAA conventions were held on the EU HTA regulation, focusing on: (i) its relevance for beating cancer; (ii) stakeholder involvement; (iii) recommended preparatory steps to ensure its successful implementation; and (iv) the role of hematology and oncology as a pacemaker for the EU HTA process. Here we summarize insights generated at the four EAA conventions about the integration of patient and clinician insights in the evolving EU HTA process, including joint scientific consultations (JSC) and joint clinical assessments (JCA).

Throughout the conventions it became clear that the interface of patient associations and clinical societies with the EU HTA process is key for successful implementation of the regulation. All involved stakeholders rely on the principles of evidence-based medicine (EBM), including best internal and external evidence, patient values and expectations, and clinical experience. It was agreed that patient and clinician perspectives on the assessments are needed to balance the technical analysis of best external evidence. While patient input is rather well defined, when and how input from clinical societies is best incorporated during the process remains unclear.

As stipulated by the EBM triad, systematic involvement of patients and clinicians throughout both JSC and JCA is key to ensuring best outcomes for patients and society as a whole, in line with the objectives of the EU HTA regulation.

The added value of medical technology is typically assessed in specific patient groups, which is less useful to guide decisions on optimal use. The magnetic resonance linear accelerator (MR-Linac) is a costly innovation that can enhance radiotherapy precision through daily imaging and plan adaptation. This study aimed to better understand its potential value and inform users on its optimal application.

Semi-structured interviews (n=24) were conducted with stakeholders, ranging from end users (radiation oncologist, clinical physicists, radiotherapy technologists, managers) to patient representatives and policy officers (e.g., procurement, health insurance, health technology assessment agency, and regulatory body). Themes explored with end users focused on the decision-making process of acquisition, current use, potential future deployment, optimal use, and the added value of MR-Linac. Themes explored with policymakers and patient representatives included their roles concerning costly medical technologies and their perspectives on the added value of the MR-Linac. Interview data were analyzed using thematic analysis.

Preliminary results showed stakeholders’ optimism for improved patient outcomes, including reduced toxicity and increased treatment efficacy. Patient representatives highlighted not only the clinical value, but also the importance of ensuring patients’ access to innovative care. End users perceived the MR-Linac as a gradual improvement, placing trust in its theoretical benefits. Additionally, they found value in its contribution to providing innovative treatment, potentially benefiting hospitals by attracting new patients. They also experienced improved job satisfaction. Policy officers emphasized the need for evidence to support potential clinical advantages as well as the significant costs associated with the MR-Linac within the broader healthcare context.

This qualitative, technology focused study deepens our understanding of the potential added value of the MR-Linac, beyond a specific context or patient group. The results not only highlight potential patient benefits, but also show a broader significance of the MR-Linac at other levels (e.g., clinician, hospital, and society). It is important to consider these different levels when guiding decisions on use of the MR-Linac.

There is currently no standardized way to share information about health technology assessment (HTA). Standardised Data on Initiatives (STARDIT) can be used to overcome current limitations in sharing data about HTA processes by providing a way to report these data. This includes which stakeholders have been involved and how, the data sources used, and any impacts or outcomes observed.

STARDIT development began in 2019, guided by participatory action research paradigms. A multidisciplinary international team of over 100 citizens, experts, and data users was involved in cocreating STARDIT. These cocreators included patients with cancer, people living with rare diseases, Indigenous Peoples from multiple countries, representatives involved in HTA processes, health researchers, environmental researchers, economists, librarians, and academic publishers. Methods used to involve people included public events, online discussions, and a public consultation process. STARDIT is free to use, and data can be submitted by anyone. Report authors can be verified to improve trust and transparency, and data can be checked for quality.

STARDIT has been used to create open access information about HTA processes that can be verified or edited by anyone at all stages of the HTA process, in multiple languages. This allows stakeholders involved in or affected by HTA processes (including patients, the public, Indigenous Peoples, and people from industry) to appraise and edit information and to self-identify the labels and terminology used to describe them. Organizations, including Australian Genomics, have recommended the use of STARDIT. Wikimedia Australia is a formal supporter and hosts data on their servers. The working beta version of STARDIT is available at ScienceForAll.World/STARDIT.

STARDIT improves access to standardized, verified information about HTA processes, enabling well-founded comparisons of the effectiveness of different HTA methods, including the most effective methods for involving stakeholders. Since STARDIT is open access and editable by anyone, it can support participatory ways of working and help improve the equity and quality of HTA processes worldwide.

Health inequalities can be described as avoidable, systematic, and unjust differences in health between different groups within society. This research described and evaluated potential methods to measure the effects of health inequalities that could be used in health technology assessment (HTA) in the UK. The research included recommendations for current and future policy objectives relating to incorporating health inequalities.

A targeted literature review was conducted to identify methodological approaches used to incorporate health inequalities in HTA. Stakeholder interviews and a workshop were conducted with a range of stakeholders in the UK. This engagement aimed to discuss any gaps in the literature and to assess whether attitudes, methods, and policies were evolving at the same rate as the literature. Other aims of the engagement included obtaining stakeholder views on health inequalities and a better understanding of the perspectives of decision-makers.

Five potential methods were identified to account for health inequalities, with equity-based weighting and distributional cost-effectiveness analysis considered to be the most feasible quantification methods. Stakeholders reiterated that a deliberative process should remain the center of HTA. Stakeholders also raised issues such as the burden on committees, trade-offs between complexity and accessibility, and the importance of measuring the size and direction of inequality impacts. Recommendations were then produced based on these findings to better account for inequalities in HTA, highlighting the importance of combining a range of approaches.

Both companies and HTA agencies should be more proactive in accounting for health inequalities. Companies should be encouraged to provide quantitative analysis on health inequalities, while decision-makers should be trained in new methods. Despite the recent rise in quantitative methods, qualitative methods remain extremely important for a layered approach to considering health inequalities.

Lower respiratory tract diseases (LRTDs) pose a public health threat, particularly among vulnerable populations. Vaccination is crucial for disease control, promoting healthy aging, and reducing mortality. However, current immunization programs for adult and the elderly are suboptimal. This project engaged healthcare professionals and citizens to develop guidelines for immunization of adults and the elderly in Italy, with a specific focus on the currently available respiratory syncytial virus (RSV) vaccination.

The project will include the following three phases: (i) Exploratory phase: a scoping review—involving a search of MEDLINE and institutional websites—to investigate current vaccinations offered to adults and the elderly in Italy for vaccine preventable LRTDs; (ii) Assessment phase: a Delphi process to identify strengths and weaknesses in the current vaccinations offered to adults and the elderly and to evaluate the latest regional and national plans for immunizing the elderly against RSV; and (iii) Policy phase: a consultation with experts to formulate recommendations for the nationwide implementation of vaccination strategies for LRTDs among adults and the elderly, paying particular attention to RSV prevention.

By analyzing current vaccinations offered in Italy, the ongoing project released new evidence and data on vaccine preventable LRTDs to enhance existing vaccination policies and strategies. This aligned a value-based decision-making process with technological innovations in vaccination. Moreover, the involvement of different stakeholders was crucial for identifying health needs and recognizing potential barriers that may hinder adequate vaccination coverage among adult and elderly populations, thus defining priority areas.

Vaccination of adult and elderly populations must be a key component of strategies aimed at promoting the maintenance of good health in the long term. The disease burden of LRTDs and RSV infections is significant and likely underestimated among the elderly. Therefore, adequate prevention strategies are crucial to reducing the national and global burden of these respiratory diseases.

As unfiltered health information overflows through social networking services, people’s health rights are being violated and unnecessary medical expenses are increasing. Citizens and patients participating in the group initiative Public Involvement in the National Evidence-based Healthcare Collaborating Agency (NECA), the sole health technology assessment (HTA) agency in South Korea, began monitoring information on health technology in social media from 2021 as a new way to identify HTA topics.

Citizens and patients in Public Involvement in NECA were provided with search keywords related to “common diseases on general public” and “diseases with high public interest”. They monitored social media platforms for one month and collected information about health technologies, such as medicines and treatments without scientific evidence on their safety and effectiveness. The target information included subjective information on health technologies based on personal experience; information with unclear sources; information that might cause excessive anxiety and fear; and information with a high risk of side effects. Information was collected from the internet and a checklist was given to the participants to evaluate the suitability of the information.

In the topic selection process, NECA, and the Korean Society of Science Journalists (KSIA) evaluated the priority of HTA topics developed from the collected information. HTA topics were evaluated using five criteria, including the possible harm the information might cause throughout society. As a result, the safety and effectiveness of saw palmetto in patients with prostatic hyperplasia and high-dose intravenous vitamin C in patients with cancer were selected. NECA conducted health technology reassessments on these topics and was able to successfully disseminate the reassessment results in collaboration with the KSIA.

Participation in the HTA process using social media lowered the barriers that laypersons experience with HTA and increased the possibility of bringing HTA topics closer to citizens’ lives. However, as there were difficulties in collecting meaningful information for developing a HTA topic, providing systematic training on health information monitoring remains a challenge.

In Singapore, health technologies were previously identified for funding consideration through horizon scanning or annual applications from clinicians, before being prioritized by the Ministry of Health advisory committees for evaluation by the Agency for Care Effectiveness (ACE). This poster describes a new process co-developed with local patient organizations to enable patients and caregivers to suggest health technologies for evaluation.

An application form was developed in plain language for patients and caregivers which requested the name of the health technology and its formulation, the medical condition it is used for, the perceived benefits and disadvantages, and the reasons why it should be funded. A factsheet explaining the selection process was codeveloped with patients, published online, and sent electronically with the application form in October 2023 to all local patient organizations, alongside the open application call for clinicians. Applications were accepted until January 2024 and then collated for prioritization in line with predefined selection criteria. All patients were notified of the outcome of their application.

Fifty applications were received from patients during the first two months of the open call compared to 75 from clinicians. Most of the patient applications (66%) requested drugs for treating asthma or respiratory conditions. Drugs requested by clinicians generally differed from those requested by patients except for 5 topics, suggesting that patients may perceive clinical need differently. Most patient applicants had used the requested drugs before and considered they were effective and convenient, but unaffordable. Health technologies were more likely to be prioritized for evaluation when their benefits were plausible and supported by evidence, and they could fill an unmet clinical need for patients.

The process will be updated in line with feedback to encourage continued patient participation annually. Enabling patients and caregivers to suggest health technologies for evaluation provides ACE with a better understanding of their needs, preferences, lived experiences, and expectations, and ensures that subsequent funding recommendations informed by ACE’s evaluations address therapeutic gaps and improve treatment affordability and patient outcomes.

In 2022, the Agency for Care Effectiveness (ACE) co-developed patient involvement processes with local patient organizations to enable patients and caregivers to share their experiential knowledge about different medical conditions and treatments to inform health technology assessments (HTAs). This presentation describes the impact of patient input on funding recommendations during the first two years of this initiative in Singapore.

A literature search was conducted to identify frameworks and indicators used by different HTA agencies that could be contextualized to evaluate local patient involvement efforts. Systematic data extraction was performed by two authors to compare patient testimonials received for HTAs conducted between May 2022 and April 2024 with key information in HTA reports, minutes from committee meetings, and published HTA guidance documents to determine the impact of patient and caregiver involvement on the committees’ deliberations and subsequent funding recommendations. The impact of patient involvement on ACE staff, existing processes, and patient participation in future HTAs was also assessed through qualitative surveys.

In the first year (May 2022 to April 2023), 112 patient responses informed 11 HTAs, while 243 responses informed 16 HTAs in the subsequent year (May 2023 to April 2024). At least one testimonial was received for 84.6 percent of HTAs in the first year, increasing to 88.9 percent in the subsequent year. Patient input addressed uncertainties in the scientific evidence and helped decision-making committees understand how different conditions affect patients and their caregivers, the outcomes that matter most to patients, and the benefits and disadvantages of different treatments. In response to feedback, ACE continually evolved its processes to meet the needs of patients and to encourage broader patient participation. Industry and patient organizations also expanded their capacity so that they can meaningfully participate in future ACE HTAs.

Continuous process improvement in response to feedback; providing patient input templates in different formats and languages to improve accessibility; and regular feedback to patient organizations on how their inputs have informed HTAs have increased patient participation, improved the legitimacy of ACE HTAs, and added value to decision-making about which health technologies should be funded in Singapore.

Patient involvement in health technology assessment (HTA) at the organizational level is vital to drive process development and capacity-building, and to ensure patients have the same opportunities as other stakeholders to effect policy changes. This presentation discusses the role and impact of the Agency for Care Effectiveness (ACE) Consumer Panel in the co-development of patient involvement and education initiatives in Singapore.

The composition and terms of reference of patient and citizen groups in overseas HTA or government agencies were reviewed to identify key aspects that could be generalized to Singapore’s context. Using selection criteria, 20 individuals from local patient or voluntary groups representing a broad range of health conditions were shortlisted; 15 were formally appointed to the Panel in April 2022. Their contributions and impact on ACE’s work and healthcare decision-making were documented throughout their two-year term. A qualitative survey was also conducted to seek members’ feedback on their participation in the Panel and identify areas to improve collaboration.

Since their appointment, ACE Consumer Panel members have played a key role in providing the collective voice of local patient organizations to guide ACE’s work and have co-developed processes that ensure meaningful patient involvement in policy development and HTA. They have also informed ACE’s workplan by providing advice on priority-setting, communication strategies, and patients’ information needs. During their term, processes to include patient input in HTAs were formalized leading to the co-development of a process guide and supporting fact sheets, a patient glossary, and two patient training modules, which will be continually updated based on patient feedback.

The ACE Consumer Panel is the first long-standing engagement of healthcare consumers by Singapore’s Ministry of Health. The Panel’s contributions and impact on ACE’s work serve as an example for other decision-makers on how to meaningfully involve patients at the organizational level, to understand their priorities and preferences, and to ensure healthcare policies remain relevant for the people affected by them.

The participation of people with mental health conditions or patient representatives is of relevance in the health technology assessment (HTA) field, although there are some challenges that need to be considered. This study was designed to gain the perspectives of patient representatives with experience participating in HTA projects, with the aim of delving into the main issues and proposing ideas for the future.

A structured interview, based on five open-ended questions, was formulated and distributed via email. Five patient representatives from the Saúde Mental FEAFES Galicia (the Federation of Associations of Family Members and Persons with Mental Illness of Galicia, Spain) who had actively engaged in HTA projects over the past five years participated in the study. The collected data were analyzed using thematic analysis.

Participants underscored the pivotal role of involving persons with mental health conditions and their representatives in HTA and emphasized the importance of knowing their perspectives, preferences, and values. Challenges included the complexity of HTA reports and processes, in terms of length and the technical language used, and socioeconomic barriers. Proposed solutions included material adaptation, streamlined processes, and institutional and professional support. Future recommendations emphasized improving awareness, disseminating the HTA field, fostering active participation, and emphasizing the relevance of participating in the HTA process. Of particular significance was the encouragement of training and capacity building among patients with mental health conditions.

This study reveals the need to strengthen and facilitate the participation of individuals with mental health conditions in HTA processes, and emphasizes the importance of knowing their perspectives, preferences, and values. Recommendations include training and capacity building, simplifying materials and processes, and providing adequate support.

Various committees provide health technology assessment (HTA) recommendations to the Australian government on health technology subsidization. All committees include consumer members, but operations vary across committees. To support the committee’s consumer members and facilitate consumer input, the Consumer Evidence and Engagement Unit (CEEU) was launched in 2019. The CEEU aims to enhance inclusion of consumer input in Commonwealth HTA processes.

The CEEU operates within the Australian Government Department of Health and Aged Care in a hybrid format. The core functions of the CEEU include secretariat support for the HTA Consumer Consultative Committee (CCC), which comprises consumer members from all HTA committees. The CEEU also summarizes consumer input for the HTA committees. In 2022, the CEEU launched the Conversations for Change series, which was developed through a range of different consultation activities. As a result, an ongoing “Changing Conversations” mandate is in place, to build and sustain relational, non-transactional partnerships in a true collaborative style.

The HTA committees and subcommittees operate across cycles with overlapping timelines, requiring consumer input to be gathered at different timepoints for different purposes. The CEEU has launched materials, including the Consultation Hub and HTA Engage, and is developing a Consultation Toolkit to raise awareness and facilitate these processes. The role of the HTA CCC is evolving as a forum to enable consumer committee members to share experiences and receive CEEU support. Improvements in consultation processes have enabled the CEEU to provide consumer comments to consumer committee members to facilitate genuine representation of consumer and other external stakeholder views.

Managing consumer input can be resource intensive, however there is increasing recognition of the importance of consumer engagement in Australian HTA. The CEEU expects continued growth and a wider remit from 2024, as new processes and changes occur. Additional materials are planned to support consumer committee members and to build capacity among consumers and consumer organizations.

Technology is central in supporting older people with their daily tasks and independence at home. This project aimed to identify technologies that can be built into residential environments (e.g., appliances, fixtures, or fittings) to support older people in activities of daily living (ADL) through a horizon scan (HS) informed by public insights on unmet needs and priorities.

A survey of members of the public was conducted to prioritize outcomes included within an evidence and gap map (EGM) framework. The EGM aimed to illustrate the current landscape of technologies supporting ADL in residential settings (e.g., care homes) and innovation gaps. The EGM results were shared with end users in a workshop discussion on the current range of technologies aimed at supporting ADL in residential settings. This was facilitated using vignettes to elicit views on unmet needs and priorities for technology development. The workshop informed the scope of the HS to identify and prioritize emerging technologies that could address unmet needs.

This project successfully embedded public involvement throughout to identify innovation gaps in technologies supporting ADL, unmet needs among end users, and potential solutions to these needs. The HS identified 190 technologies that were ready to market. All the technologies had potential to address identified unmet needs and could be built into the residential environment to support older people with ADL and to improve their quality of life, independence, and safety at home. Horizon scanning research can meaningfully involve stakeholders and take direction from their insights to enable voices less often heard to drive innovation in areas where it is needed.

Involving stakeholders in research using evidence synthesis and qualitative methods helps to gain a better understanding of gaps in innovation, the related unmet needs, and the technologies that might address these needs. Public involvement in the survey and workshop influenced the conduct and interpretation of the EGM, the scope of the HS, and the interpretation of the findings.

Ultrarare diseases (URD) represent a challenge to health technology assessment (HTA). The traditional framework for assessing efficacy and cost effectiveness may be biased to include clinically relevant outcomes, leaving patient-centered outcomes doomed to neglect. Here we explore patient-centered outcomes in the context of patient and citizen involvement in the assessment of URD by the Brazilian National Committee for Health Technology Incorporation (CONITEC).

We assessed 53 HTA reports from CONITEC that evaluated URD-related technologies (and included highlights of patients’ and citizens’ perspectives during recommendation meetings) published from 2012 to 2022. Data extraction was performed by two independent researchers. Data on year of report, sex, ethnicity, category (patient or family), and previous experience with the assessed technology were extracted and analyzed using descriptive statistics. Patients’ and citizens’ narratives were collated from the reports. A thematic analysis was conducted according to patient-centered outcomes and technology-related outcomes and was then compared with the evidence synthesis protocol described in the HTA.

Only seven URD-related HTA reports registered patient or citizen participation, all of which were published in 2022. The age of two participants was reported (both 17 years). Six participants were women. Ethnicity was not reported. All participants had previous experience with the technology. Four participants were family or caregivers and three were patients. Considering patient-centered outcomes, physical (muscular strength) and emotional (self-confidence) improvements that positively affected independence in basic daily functions were reported. These functions included activities such as dressing, self-care, cooking, and leisure. Advantages listed for the assessed technologies included the possibility of self-administration of medication (e.g., swallowing a pill, opening a medicine bottle, and using a syringe).

The results show that although, in some cases, primary outcomes reported in evidence synthesis protocols include patient-centered outcomes (e.g., activities of daily living), in other cases the evidence synthesis failed to identify relevant studies. In other cases, the reports failed to differentiate between primary and secondary outcomes or to fully account for patient-centered outcomes.

The Scottish Medicines Consortium (SMC) patient group submission process includes a written submission of patient experience evidence from Patient Group Partners (PGPs). To maximize the relevance and quality of information included in submissions, the first draft is reviewed by the Public Involvement Team and feedback is provided. The submission is then amended by the submitting PGP before final submission.

Upon receiving a new written patient group submission, the Public Involvement Team reviews it and provides feedback highlighting any evidence gaps and areas where wording could be amended to strengthen the patient voice. The draft submission is then amended by the submitting PGP, with support from the SMC reviewer. Once finalized, the submission is collated into the body of evidence used by the SMC Committee members to assess a new medicine. The satisfaction of participating PGPs is continually assessed by an online survey. During the 2021 to 2023 period, the SMC received 232 patient group submissions, with 77 percent being amended and strengthened after receiving feedback.

The feedback and collaboration on amendments to draft submissions has improved the quality of patient experience evidence submitted to the SMC by PGPs. PGPs value their draft evidence being reviewed by the SMC Public Involvement Team prior to final submission. This approach resulted in high levels of trust in and satisfaction with how the SMC involves patient representatives in medicine assessments. In 2022, 89 percent of the 28 PGPs surveyed were very satisfied and 11 percent were satisfied with the support provided during the submission process.

Evaluation of the SMC’s formal process to provide input and feedback on draft written patient group submissions demonstrates high levels of satisfaction with how the SMC works in partnership with PGPs. This approach has strengthened written evidence of patient experiences for SMC assessments. Furthermore, it helps to build and maintain a partnership approach in how the SMC works with PGPs.